Home › Spinal Muscular Atrophy › NCT07287982
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
A Phase 2 Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Who can join
Ages 5 to 17 · All sexes
Full eligibility criteria
Inclusion Criteria: * Is aged ≥5 to \<18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having a parent or guardian sign the ICF, and can comply with protocol * requirements. * Has documented historical genetic diagnosis of 5q-SMA. * Currently receiving a stable SMA treatment regimen (nusinersen or risdiplam) and/or have a history of onasemnogene abeparvovec treatment * Must be able to walk at least 50 meters without walking aids in the 6MWT at screening Exclusion Criteria: * Known medical condition that would interfere with an accurate assessment of SMA, confound the results of the study, or put the participant at undue risk, as assessed by the investigator * Recent major surgery, except spinal fusion, within 3 months of screening or intends to have major surgery during the study * Current or previous administration of antimyostatin therapies in the past 6 months * Severe scoliosis (defined as curvature \>40°) and/or contractures at screening. o History of spinal fusion within 6 months before screening or planned during the study * Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for daytime treatment while awake. Ventilation used overnight or during daytime naps is acceptable.
About the study
This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it. The study consists of a double-blinded treatment period (DBTP) where participants will either receive ARGX-119 IV or placebo IV, in addition to disease-modifying therapy (DMT) for 24 weeks. Participants who complete the DBTP will enter the open-label active-treatment extension period (ATEP) during which all participants will receive ARGX-119 IV up to 100 weeks (approximately 2 years).
What is being tested
- ARGX-119 IV (biological)
- Placebo IV (other)
Sponsor: argenx · Participants: 60 · Started: Dec 19, 2025
Contact the study team
- Sabine Coppieters, MD · Phone: 857-350-4834
Official record on ClinicalTrials.gov — NCT07287982
Locations in the U.S.
| Arkansas | Arkansas Children's Hospital, Little Rock |
| California | Stanford University Medical Center, Stanford |
| Connecticut | Connecticut Children's Medical Center, Hartford |
| Florida | Rare Disease Research FL LLC, Kissimmee |
| Illinois | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago |
| Iowa | University of Iowa Stead Family Children's Hospital, Iowa City |
| Maryland | The Johns Hopkins Hospital, Baltimore (Not yet recruiting) |
| Massachusetts | Boston Children's Hospital, Boston |
| Missouri | The Curators of the University of Missouri on behalf of University of Missouri Health Care, Columbia (Not yet recruiting) |
| New York | Columbia University Herbert Irving Comprehensive Cancer Center, New York (Not yet recruiting) |
| North Carolina | Rare Disease Research NC, LLC, Hillsborough |
| Pennsylvania | Children's Hospital Philadelphia - Neurology, Philadelphia (Not yet recruiting) |
| Texas | Neurology Rare Disease Center, Flower Mound Texas Children's Hospital, Houston |
| Virginia | Childrens Hospital of The Kings Daughters, Norfolk (Not yet recruiting) |
Conditions
From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.